Taiwan's National Health Insurance (NHI) system has approved its first-ever patient to receive a one-time NT$49 million (US$1.53 million) drug for spinal muscular atrophy (SMA), which was added to a list of covered treatments earlier this month, an official said Monday.
The patient, a baby less than 6 months old from southern Taiwan, received approval for the gene therapy drug marketed as Zolgensma on Aug. 8, following an application by their hospital, National Health Insurance Administration (NHIA) Deputy Director-general Tsai Shu-ling (蔡淑鈴) told CNA.
The child is the first applicant in Taiwan to be given approval to receive the drug, out of an anticipated eight such patients per year, Tsai said.
After receiving the treatment, the child will have to have their muscular fitness tested every four months and undergo a general health assessment every year, Tsai said.
The child's condition will also have to be monitored for at least 10 years, due to the lack of long-term data on the relatively new drug, she added.
Firsts for NHI
The drug's NT$49 million cost will be paid in annual installments to the manufacturer -- also a first for the NHI -- on the condition that it effectively treats SMA, Tsai said.
Upon approving coverage of Zolgensma earlier this year, the NHIA said it had managed to negotiate the price of the drug to NT$49 million when purchased for use in Taiwan to treat SMA, a rare neuromuscular disorder.
It is reported to have a list price of US$2.1 million in the United States.
The approval marked the first time the NHI system has covered a gene therapy -- though its coverage is limited to infants aged 6 months and below who develop a genetic mutation for the disorder.
On Monday, the NHIA said it is considering adding SMA to a list of conditions for which newborns are screened without charge. The self-paid test currently costs NT$400.
SMA is a rare neuromuscular genetic disease that affects the central nervous system, peripheral nervous system and voluntary muscle movements in patients, which results in the degeneration of muscles in limbs and respiratory organs.
